PMID- 35982335
OWN - NLM
STAT- MEDLINE
DCOM- 20221018
LR  - 20221102
IS  - 1432-1912 (Electronic)
IS  - 0028-1298 (Print)
IS  - 0028-1298 (Linking)
VI  - 395
IP  - 11
DP  - 2022 Nov
TI  - Efficacy and toxicity of hydroxyurea in mast cell activation syndrome patients 
      refractory to standard medical therapy: retrospective case series.
PG  - 1441-1447
LID - 10.1007/s00210-022-02282-8 [doi]
AB  - Determine efficacy and adverse events (AEs) of hydroxyurea (HU) in mast cell 
      activation syndrome (MCAS) patients who were refractory to standard medical 
      therapy. An electronic chart review was performed to find MCAS patients who 
      received HU in a MCAS medical practice. Diagnosis of MCAS was established on the 
      basis of mast cell (MC) activation symptoms in >/= 5 systems plus >/= 1 abnormal MC 
      mediators and/or >/= 20 MC/high power field on duodenal biopsies. Medicines not 
      providing significant clinical improvement prior to HU were tabulated. The 
      following symptoms were evaluated by patients on a 0-10 scale prior to and at the 
      study conclusion: bone pain, abdominal pain, diarrhea, bloating, and nausea. 
      Safety labs were obtained on a regular basis. Twenty out of three hundred ten 
      (8.4%) MCAS patients received HU. Patients included 22 females, average age 
      42.4 years. Dysautonomia was present in 60%. An average of 10.6 (SD 1.7, range 
      8-13) medications were used prior to adding HU to various concomitant 
      medications. Average dose of HU was 634 mg. In 20 patients who continued therapy 
      for >/= 2 months, there was statistically significant reduction of bone pain, 
      abdominal pain, diarrhea, bloating, and nausea. Fourteen patients noted prolonged 
      success with therapy. Six patients stopped HU within 6 weeks owing to AEs. Four 
      patients treated >/= 2 months had AEs and 2 led to HU cessation. All AEs were 
      reversible. Refractory MCAS patients showed clear significant improvement in bone 
      pain and gastrointestinal symptoms on HU. Systematic monitoring was effective in 
      preventing the occurrence of severe HU-induced adverse events.
CI  - (c) 2022. The Author(s).
FAU - Weinstock, Leonard B
AU  - Weinstock LB
AD  - Clinical Medicine, Department of Medicine, Washington University School of 
      Medicine, President, Specialists in Gastroenterology, 11525 Olde Cabin Road, St. 
      Louis, MO, 63141, USA.
FAU - Brook, Jill B
AU  - Brook JB
AD  - , Truckee, USA.
FAU - Molderings, Gerhard J
AU  - Molderings GJ
AD  - Institute of Human Genetics, University Hospital Bonn, 53127, Bonn, Germany. 
      molderings@uni-bonn.de.
LA  - eng
PT  - Journal Article
DEP - 20220819
PL  - Germany
TA  - Naunyn Schmiedebergs Arch Pharmacol
JT  - Naunyn-Schmiedeberg's archives of pharmacology
JID - 0326264
RN  - X6Q56QN5QC (Hydroxyurea)
SB  - IM
MH  - Abdominal Pain/chemically induced/drug therapy
MH  - Adult
MH  - Diarrhea/chemically induced/drug therapy
MH  - Female
MH  - Humans
MH  - *Hydroxyurea/adverse effects
MH  - *Mast Cell Activation Syndrome
MH  - Mast Cells
MH  - Nausea/chemically induced/drug therapy
MH  - Retrospective Studies
PMC - PMC9388361
OTO - NOTNLM
OT  - Efficacy
OT  - Hydroxyurea
OT  - Mast cell activation syndrome
OT  - Toxicity
OT  - Treatment
COIS- Dr. Weinstock is an uncompensated, volunteer medical advisor to the startup 
      company MC Sciences, Ltd. Dr. Weinstock is a compensated medical advisor to 
      Statera BioPharma. Mrs. Brook has no disclosures. Dr. Molderings is the 
      co-founder and chief medical officer of the startup company MC Sciences, Ltd.
EDAT- 2022/08/19 06:00
MHDA- 2022/10/19 06:00
PMCR- 2022/08/19
CRDT- 2022/08/18 23:34
PHST- 2022/04/13 00:00 [received]
PHST- 2022/08/11 00:00 [accepted]
PHST- 2022/08/19 06:00 [pubmed]
PHST- 2022/10/19 06:00 [medline]
PHST- 2022/08/18 23:34 [entrez]
PHST- 2022/08/19 00:00 [pmc-release]
AID - 10.1007/s00210-022-02282-8 [pii]
AID - 2282 [pii]
AID - 10.1007/s00210-022-02282-8 [doi]
PST - ppublish
SO  - Naunyn Schmiedebergs Arch Pharmacol. 2022 Nov;395(11):1441-1447. doi: 
      10.1007/s00210-022-02282-8. Epub 2022 Aug 19.